Article
Biochemical Research Methods
Stefan J. Tekel, Nicholas Brookhouser, Kylie Standage-Beier, Xiao Wang, David A. Brafman
Summary: The introduction of transient reporters of editing enrichment (TREE) has enabled highly efficient single-base editing of human cells using a transient episomal fluorescent reporter, allowing for rapid generation of clonal editing efficiencies exceeding 80% in biallelic or multiplexed edited isogenic human pluripotent stem cell lines within approximately 3-4 weeks.
Review
Agriculture, Dairy & Animal Science
Julia Popova, Victoria Bets, Elena Kozhevnikova
Summary: Genome editing has practical applications in farm animals, improving production traits, economic value, and disease resistance. It also has potential in biomedical research and drug production, as well as xenograft donors. Recent advancements in site-specific nucleases and embryological delivery methods have revolutionized transgenesis, providing efficient and reliable tools for genome engineering in agriculture.
Review
Biochemistry & Molecular Biology
Yulin Mu, Chengxiao Zhang, Taihua Li, Feng-Jie Jin, Yun-Ju Sung, Hee-Mock Oh, Hyung-Gwan Lee, Long Jin
Summary: Lactobacillus, important in food production and probiotics, can benefit from CRISPR/Cas9-based genome editing to improve strain efficiency and achieve traceless genome modification.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Multidisciplinary Sciences
Dusko Lainscek, Vida Forstneric, Veronika Mikolic, Spela Malensek, Peter Pecan, Mojca Bencina, Matjaz Sever, Helena Podgornik, Roman Jerala
Summary: The CRISPR/Cas system, when coupled with Exonuclease III, can enhance gene editing efficiency and lead to significant gene knock-out effects, especially in cell lines, primary cells, and in vivo. This approach has great potential for treating diseases with a genetic etiology.
NATURE COMMUNICATIONS
(2022)
Article
Microbiology
Xueli Zhang, Chaohui Zhang, Caijiao Liang, Bizhou Li, Fanmei Meng, Yuncan Ai
Summary: Bacteriophages, the most abundant organisms in the biosphere, have been sequenced extensively. However, the study of bacteriophage functional genomics has been hindered by a lack of effective research methods. This study designed a phage genome editing platform based on the CRISPR-Cas9 system, and successfully achieved gene editing in Vibrio natriegens phage TT4P2. This platform has the potential to advance research on phage gene diversity and accelerate the development of phage synthetic biology and nanotechnology.
MICROBIOLOGY SPECTRUM
(2022)
Review
Pharmacology & Pharmacy
Siwei Chen, Deng Chen, Bin Liu, Hidde J. Haisma
Summary: CRISPR/Cas9-mediated genome engineering has extensive applications in basic biology, biotechnology, and medicine. However, the low gene modification efficiency and uncontrollable prolonged Cas9 activity hinder its further use. Researchers have explored small molecules with clinical potential to precisely modulate CRISPR/Cas9 genome editing activity.
DRUG DISCOVERY TODAY
(2022)
Article
Materials Science, Multidisciplinary
Yue Wang, Xiujue Zheng, Jun Liu, Qixian Chen, Yan Zhao
Summary: The emergence of CRISPR-based genome editing systems has revolutionized the field of molecular biology and holds great promise for the treatment of various diseases. However, the lack of suitable delivery vehicles for effective intracellular delivery of CRISPR components limits its practical applications. In this study, a nanometer-scaled vesicle platform (CRISPRsome) was developed to efficiently deliver the essential components of the CRISPR system, resulting in significant gene knockout efficacy in cancer cells. This study highlights the potential of CRISPRsome in facilitating the use of CRISPR technology in practical applications.
ACS MATERIALS LETTERS
(2022)
Review
Biochemistry & Molecular Biology
Min Ho Lee, Jae Il Shin, Jae Won Yang, Keum Hwa Lee, Do Hyeon Cha, Jun Beom Hong, Yeoeun Park, Eugene Choi, Kalthoum Tizaoui, Ai Koyanagi, Louis Jacob, Seoyeon Park, Ji Hong Kim, Lee Smith
Summary: This review highlights the current and potential use of CRISPR-Cas9 in the management of autoimmune diseases. Several studies have shown that CRISPR-Cas9 can be utilized for immunomodulation, reducing cholesterol, and treating rare diseases. However, there is limited research on the treatment of autoimmune diseases using CRISPR-Cas9.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biotechnology & Applied Microbiology
Kristina J. Tatiossian, Robert D. E. Clark, Chun Huang, Matthew E. Thornton, Brendan H. Grubbs, Paula M. Cannon
Summary: This study found that CRISPR-Cas9 indel signatures can be used to identify gRNAs that maximize HDR outcomes, with the frequency of deletions resulting from MMEJ repair of at least 3 base pairs better predicts HDR frequency. Existing tools can be repurposed to identify gRNAs that promote HDR.
Article
Plant Sciences
Syed Inzimam Ul Haq, Dianfeng Zheng, Naijie Feng, Xingyu Jiang, Feng Qiao, Jin-Sheng He, Quan-Sheng Qiu
Summary: This review summarizes the advances of CRISPR/Cas9 technology in plant genome editing and discusses its applications in forage breeding, as well as the potential and challenges.
JOURNAL OF PLANT PHYSIOLOGY
(2022)
Article
Chemistry, Physical
Juhee Lee, Yoo Kyung Kang, Eonju Oh, Juhee Jeong, San Hae Im, Duk Ki Kim, Haeshin Lee, Sang-Gyu Kim, Keehoon Jung, Hyun Jung Chung
Summary: The study presents a cancer gene therapy strategy based on NanoRNP that efficiently blocks the PD-L1 immune checkpoint and induces an antitumor effect in vivo without the need for combination therapy. In vivo results demonstrate that NanoRNP can induce indels in target cells at high frequencies, significantly suppressing tumor growth.
CHEMISTRY OF MATERIALS
(2022)
Article
Multidisciplinary Sciences
Beate Rieblinger, Hicham Sid, Denise Duda, Tarik Bozoglu, Romina Klinger, Antonina Schlickenrieder, Kamila Lengyel, Krzysztof Flisikowski, Tatiana Flisikowska, Nina Simm, Alessandro Grodziecki, Carolin Perleberg, Andrea Bahr, Lucie Carrier, Mayuko Kurome, Valeri Zakhartchenko, Barbara Kessler, Eckhard Wolf, Lutz Kettler, Harald Luksch, Ibrahim T. Hagag, Daniel Wise, Jim Kaufman, Benedikt B. Kaufer, Christian Kupatt, Angelika Schnieke, Benjamin Schusser
Summary: Research on genetically modified animals has mostly focused on mice, but also includes species like pigs that are more physiologically similar to humans, and cross-species comparisons with phylogenetically distant species like chickens. CRISPR-Cas9 is a versatile genetic editing method applicable across various species. The successful generation of transgenic chickens and pigs that express Cas9 in all organs has confirmed the functionality of Cas9 across different target genes, cell types, and in vivo applications.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2021)
Article
Biochemistry & Molecular Biology
Guoxu Song, Fei Zhang, Chunhong Tian, Xing Gao, Xiaoxiao Zhu, Dongdong Fan, Yong Tian
Summary: This study identified novel anti-CRISPR families that have the ability to inhibit Cas9 activity and even control genome editing post-translationally. By studying different Acr variants and developing chemically inducible control, new avenues for CRISPR-Cas9 applications have been established.
NUCLEIC ACIDS RESEARCH
(2022)
Article
Multidisciplinary Sciences
J. Boutin, J. Rosier, D. Cappellen, F. Prat, J. Toutain, P. Pennamen, J. Bouron, C. Rooryck, J. P. Merlio, I. Lamrissi-Garcia, G. Cullot, S. Amintas, V. Guyonnet-Duperat, C. Ged, J. M. Blouin, E. Richard, S. Dabernat, F. Moreau-Gaudry, A. Bedel
Summary: The study reveals the genotoxicity caused by CRISPR-Cas9 leading to loss of heterozygosity changes, while also highlighting the potential safety concerns of this technology for gene therapy.
NATURE COMMUNICATIONS
(2021)
Review
Biochemistry & Molecular Biology
Chao Zhang, Shanhe Liu, Xuan Li, Ruixuan Zhang, Jun Li
Summary: CRISPR/Cas-based genome editing technologies have revolutionized plant science by allowing precise manipulation of plant genomes. Virus-induced genome editing (VIGE) has emerged as a powerful method for delivering CRISPR/Cas reagents into plant cells, with high editing efficiency and simplified process. This article provides an overview of CRISPR/Cas-based genome editing and focuses on the VIGE systems, types of viruses used for CRISPR/Cas9 cassette delivery, recent applications, and challenges and potential of VIGE in plants.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Endocrinology & Metabolism
Elina Leena Harjunen, Minna Laine, Ritva Tikkanen, Paivi Helenius
JOURNAL OF INHERITED METABOLIC DISEASE
(2020)
Article
Clinical Neurology
A. Tokola, M. Laine, R. Tikkanen, T. Autti
AMERICAN JOURNAL OF NEURORADIOLOGY
(2019)
Review
Cell Biology
Miroslava Didiasova, Antje Banning, Heiko Brennenstuhl, Sabine Jung-Klawitter, Claudio Cinquemani, Thomas Opladen, Ritva Tikkanen
Article
Neurosciences
Viljami Sairanen, Anna Tokola, Ritva Tikkanen, Minna Laine, Taina Autti
Article
Cell Biology
Antje Banning, Anna Zakrzewicz, Xin Chen, Steven J. Gray, Ritva Tikkanen
Summary: The study established human cell lines (HEK293T and HeLa) as permissive for AAV9 transduction by knocking out a CMP-sialic acid transporter, providing a model system for establishing potency assays for AAV9-based gene therapy for human diseases.
Article
Dermatology
Christoph Hudemann, Roberto Maglie, Maria Llamazares-Prada, Benedikt Beckert, Dario Didona, Ritva Tikkanen, Thomas Schmitt, Takashi Hashimoto, Jens Waschke, Michael Hertl, Ruediger Eming
Summary: This study suggests that anti-DSC3 IgG in a mouse model of pemphigus can cause acantholysis through the activation of p38 MAPKs and EGFR. Additionally, the induction of anti-human DSC3 IgG is associated with DSC3-specific T cells in association with HLA-DRB1*04:02.
JOURNAL OF INVESTIGATIVE DERMATOLOGY
(2022)
Article
Cell Biology
Antje Banning, Ritva Tikkanen
Summary: Splicing defects caused by mutations in consensus sequences are common in human diseases and often lead to loss of protein function. Modulation of splicing by small molecules may be an effective therapy for genetic diseases.
Article
Cell Biology
Ada-Sophia Clees, Verena Stolp, Bjoern Haeupl, Dominik C. Fuhrmann, Frank Wempe, Marcel Seibert, Sarah Weber, Antje Banning, Ritva Tikkanen, Richard Williams, Bernhard Bruene, Hubert Serve, Frank Schnuetgen, Ivana von Metzler, Nina Kurrle
Summary: The study analyzed the adaptation of multiple myeloma cells to hypoxia, revealing changes in protein expression and regulation of specific proteins under hypoxic conditions, including the cysteine protease LGMN, which can impact the growth of multiple myeloma cells in hypoxic environments.
Editorial Material
Cell Biology
Ritva Tikkanen
Article
Cell Biology
Anna Zakrzewicz, Celina Wuerth, Benedikt Beckert, Simon Feldhoff, Katrien Vanderheyden, Stian Foss, Jan Terje Andersen, Hans de Haard, Peter Verheesen, Vladimir Bobkov, Ritva Tikkanen
Summary: Blocking FcRn can stabilize keratinocyte monolayer and provide a novel treatment strategy for pemphigus.
Review
Oncology
Valentina Fogazzi, Marcel Kapahnke, Alessandra Cataldo, Ilaria Plantamura, Elda Tagliabue, Serena Di Cosimo, Giulia Cosentino, Marilena V. Iorio
Summary: Breast cancer is the most commonly diagnosed malignancy in women globally, with 20% of cases being HER2 positive. Despite the improvement in prognosis with anti-HER2 agents, a significant number of patients do not respond to treatment. MicroRNAs are promising clinical tools for managing HER2 positive breast cancers due to their detectability and stability in tissues and blood, as well as their potential as therapeutic agents, prognostic and predictive biomarkers.
Article
Neurosciences
Arttu Ruohola, Eero Salli, Timo Roine, Anna Tokola, Minna Laine, Ritva Tikkanen, Sauli Savolainen, Taina Autti
Summary: Magnetic resonance imaging data can be used to develop computer-assisted diagnostic tools for neurodegenerative diseases. This study found that features extracted from different types of magnetic resonance images can effectively differentiate AGU patients from healthy controls.
Article
Biochemistry & Molecular Biology
Antje Banning, Minna Laine, Ritva Tikkanen
Summary: Novel treatment strategies for rare genetic diseases require reliable biomarkers to assess treatment effect. In this study, a validated fluorometric AGA activity assay was established for assessing AGA activity in human serum samples. The assay proved to be suitable for diagnosing AGU and potentially monitoring treatment effect.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Endocrinology & Metabolism
Miroslava Didiasova, Antje Banning, Ritva Tikkanen
Summary: Due to the low number of patients, rare genetic diseases pose a special challenge for therapy development. Precision medicine uses specific molecular information to elucidate effective therapy possibilities for patient-specific pathogenic variants. This review focuses on personalized precision medicine approaches for cost-effective and fast therapy development, including drug repurposing and suitable cell culture models.
JOURNAL OF INHERITED METABOLIC DISEASE
(2023)
Article
Biochemistry & Molecular Biology
Heiko Brennenstuhl, Miroslava Didiasova, Birgit Assmann, Mariarita Bertoldi, Gianluca Molla, Sabine Jung-Klawitter, Oya Kuseyri Huebschmann, Julian Schroter, Thomas Opladen, Ritva Tikkanen
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2020)